Down Syndrome Clinical Trials - Study of Alzheimer's Disease in Down Syndrome
- Conditions
- Alzheimer's Disease in Down Syndrome
- Registration Number
- NCT04149197
- Lead Sponsor
- LuMind IDSC Foundation
- Brief Summary
The primary objective is to characterize trajectories of change on the primary outcome measures in this study population through longitudinal collection of measures of cognition, function, behavior, and health status.
- Detailed Description
The DS-CTN will engage in rigorous, high quality research to better understand AD-DS and the medical needs of this population. The LIFE-DSR study will utilize the DS-CTN network to achieve these goals through evidence-based research and a combination of proven and novel methodologies. The network will initially recruit and employ experts and clinical trial sites who have access to adults with DS and whose focus is on AD-DS.
This trial also aims to develop sensitive and well-validated assessment instruments of cognition, behavior and function appropriate for future clinical trials directed at AD-DS in adults with DS ages 25 and older.
In addition, this LIFE-DSR study will serve as a parent protocol that includes one or more sub-studies, each of which has its own protocol and consent form. These sub-studies will recruit from eligible participants enrolled in this LIFE-DSR protocol.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 252
- Age 25 years or older
- Diagnosis of DS, typically supported by karyotype analysis documenting full trisomy for chromosome 21 or complete unbalanced translocation of chromosome 21. Karyotype analysis is not required for study entry
- Participants, or Legal Authorized Representative, and their study partner if applicable, in the opinion of the investigator, are able to understand and willing to sign written informed consent.
- Participants must have a study partner who has frequent interaction with the participant on a regular basis, will agree to participate in annual clinic visits, can provide accurate responses to questions about the participant, and facilitate participation in the study visits, in the opinion of site PI or study coordinator.
- Participant and study partner must be capable of reliably completing study assessments.
Exclusion Criteria Participants and study partners who, in the opinion of the site PI, are not able to complete trial procedures or adhere to the schedule of study assessments will be excluded from study participation.
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Cognitive Measure through study completion, an average of 2 years Severe Impairment Battery (SIB) with Shoebox Test. If the participant achieves a score 60 or above on the SIB, the Down Syndrome Mental Status Examination (DS-MSE) will also be administered. Subscale Ranges:
Social Interaction - 0 - 6 Orientation - 0 - 8 Visuospatial Ability - 0 - 8 Construction - 0 - 12 Language - 0 - 56 Memory - 0 - 16 Praxis - 0 - 8 Attention - 0 - 15 Orienting to name - 0 - 4
Higher scores reflect better performance for each subscale. Scores above 60 mean the participant completes the Shoebox Memory Test.Behavioral Measure through study completion, an average of 2 years Neuropsychiatric Inventory (NPI) - Neuropsychiatric Inventory (NPI) evaluates both the frequency and severity of 10 neuropsychiatric features, including delusions, hallucinations, agitation/aggression, dysphoria, anxiety, euphoria, apathy, disinhibition, irritability and lability, and aberrant motor behavior, as well as evaluates sleep and appetite/eating disorders. Frequency assessments range from 1 (occasionally, less than once per week) to 4 (very frequently, once or more per day or continuously). Severity assessments range from 1 (mild) to 3 (severe). The score for each subscale is the product of severity and frequency and the total score is the sum of all subscales.
Functional Measure through study completion, an average of 2 years The Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Vineland-3 is an adaptive behavior measure used to assess intellectual/developmental/other disabilities. Total score range: 0 - 140
Expressive Communication: 0 - 98 49 questions - range: 0 - 2 per Q Written Communication: 0 - 76 38 questions - range: 0 - 2 per Q Personal Daily Living Skills - 0 - 110 55 questions - range: 0 - 2 per QHealth Measures through study completion, an average of 2 years New-onset seizures or significantly increased frequency of seizures
- Secondary Outcome Measures
Name Time Method Exploratory Outcome Measure Preliminary composite measure from the scales being used in LIFE-DSR through study completion, an average of 2 years To derive a preliminary composite measure from the scales being used in LIFE-DSR that is most sensitive to change in this population and which can be validated in a future prospective study. If the participant achieves a score 60 or above on the SIB, the Down Syndrome Mental Status Examination (DS-MSE) will also be administered. The maximum score is 100.
Exploratory Outcome Measure Novel multi-domain instrument for AD-DS development through study completion, an average of 2 years To integrate and further validate a novel and highly sensitive multidomain instrument for AD-DS upon its completion (Professor A. Strydom, Kings College, London). This instrument is being developed under a separate protocol and upon its validation will be integrated into LIFE-DSR to evaluate its responsiveness to change in clinically important domains.
Exploratory Outcome Measure Potential Screening Measures through study completion, an average of 2 years Individual subscales from the larger test batteries included in the LIFE-DSR Primary Outcome Measures, Severe Impairment Battery (SIB) with Shoebox Test, the Down Syndrome Mental Status Examination (DS-MSE), NPI, and Vineland3. These could then be further validated in a future prospective study.
SIB - Social Interaction - 0 - 6 Orientation - 0 - 8 Visuospatial Ability - 0 - 8 Construction - 0 - 12 Language - 0 - 56 Memory - 0 - 16 Praxis - 0 - 8 Attention - 0 - 15 Orienting to name - 0 - 4
Scores above 60 mean the participant completes the Shoebox Memory Test.
Vineland-3 -Total score range: 0 - 140
DLD - Questions with a possible range of 0 - 2 per question. Higher scores reflect worse performance.
NPI - The score for each subscale is the product of severity and frequency and the total score is the sum of all subscales.
Trial Locations
- Locations (13)
University California Irvine
๐บ๐ธIrvine, California, United States
Rush University Medical Center
๐บ๐ธChicago, Illinois, United States
Emory University
๐บ๐ธAtlanta, Georgia, United States
University of California, San Diego
๐บ๐ธLa Jolla, California, United States
Advocate Health
๐บ๐ธPark Ridge, Illinois, United States
Kansas University Medical Center
๐บ๐ธFairway, Kansas, United States
University Of Kentucky
๐บ๐ธLexington, Kentucky, United States
Kennedy Krieger Institute
๐บ๐ธBaltimore, Maryland, United States
Massachusetts General Hospital
๐บ๐ธCharlestown, Massachusetts, United States
Case Western Reserve University
๐บ๐ธCleveland, Ohio, United States
Duke University Medical Center
๐บ๐ธDurham, North Carolina, United States
Cincinnati Children's Hospital Medical Center
๐บ๐ธCincinnati, Ohio, United States
Barrow Neurological Institute
๐บ๐ธPhoenix, Arizona, United States